An estimated 25-30 million Americans suffer from rare disorders, which are defined in the Orphan Drug Act as having prevalence of 200,000 or less. Hundreds of millions, primarily in developing countries, have relatively common medical problems for which there are few, if any, effective therapies. These have been dubbed neglected diseases because most have been ignored by governmental agencies and the biomedical industry. Some neglected diseases such as malaria and tuberculosis are also a growing public health concern in the United States. Together rare and neglected diseases cause enormous suffering and exact a very high cost to society in terms of medical expenses and lost productivity. Biomedical research is providing scientists with a greater understanding of rare diseases and the potential targets that can be exploited for development of new therapies, known as of orphan drugs and biologics. Industry, however, has been reluctant to capitalize on these opportunities because of the high drug development failure rate and the small markets associated with rare diseases. In an effort to address this dilemma, governmental and non-governmental organizations are seeking to attract academic scientists to orphan drug research. A global, collaborative effort among scientists is needed to fully leverage the potential to develop new therapies for rare diseases. The proposed conference is designed to bring together a wide range of translational and clinical researchers from India and the US with interests in rare diseases and orphan drugs to share their research and foster collaborations. PUBLIC HEALTH RELEVANCE: A three day conference entitled "Indo-US Conference on Rare Diseases and Orphan Drugs" will allow attendees to share their research and expertise, learn about NIH and FDA programs, and gain a better understanding of the commercialization and regulatory issues related to the orphan drug development. Conference outcomes include: 1) a better understanding of research opportunities in rare and neglected diseases, 2) establishment of new international collaborations, and 3) initiation of orphan drug discovery and development in developing countries such as India. The conference will serve as a model for future conferences intended to accelerate the worldwide development of new therapies for rare and neglected medical problems. Rare and neglected diseases are a major, worldwide public issue. Millions in the US and hundreds of millions worldwide suffer from disorders for which there are few, if any, safe and effective therapies. The Indo-US Research Conference on Rare Diseases and Orphan Drugs will bring together leading scientists from India and the US to share research, build collaborations, and increase awareness of the need for the development of orphan drugs and biologics particularly in developing countries.